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Hyperactive Factor IX Padua: A Game-Changer for Hemophilia Gene Therapy

21 Dec 2017

Abstract excerpt

For the past 25 years, the development of gene therapy for hemophilia has fueled technological innovations and led to emerging insights that benefited the field at large.1,2 It was particularly encouraging that sustained expression of coagulation factor IX (FIX) was achieved after liver-directed gene therapy with adeno-associated viral vectors (AAVs) in patients with severe hemophilia B.3 Neverthless, the...

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PMID: 29274719
DOI: 10.1016/j.ymthe.2017.12.007

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